Sirna Therapeutics, Inc. Licenses Worldwide Exclusive Rights To microRNA Technology From University Of Massachusetts Medical School

SAN FRANCISCO, May 10 /PRNewswire-FirstCall/ -- Sirna Therapeutics, Inc. a leading RNAi therapeutics company, announced today that it has signed an exclusive worldwide licensing agreement with the University of Massachusetts Medical School (UMMS) for the rights to patents covering microRNA (miRNA) technology for the modulation of gene expression. MicroRNA is involved in the RNA interference (RNAi) mechanism and can play a critical role in gene silencing. Blocking the function of miRNAs holds significant potential for the treatment of human disease. In addition to the modulation of gene expression by blocking miRNA function, miRNAs on their own can be used as therapeutic agents.

Like short interfering RNAs (siRNAs), miRNAs are involved in the RNA interference (RNAi) mechanism. However, while siRNAs direct the cleavage of messenger RNA (mRNA) synthesized by a gene, microRNAs appear to predominantly block translation of proteins by binding to the mRNA. The mechanism by which siRNAs and miRNAs induce gene silencing are complementary to one another, thereby presenting a dual approach to harnessing the RNAi mechanism to down regulate pathogenic proteins and viruses.

"Exclusive license to these Zamore miRNA patents, combined with Sirna's existing intellectual property on miRNA, gives our Company a leading patent position in the emerging area of miRNA technology and use of miRNA as therapeutic agents or targets," said Bharat Chowrira, Ph.D., Vice President, Legal Affairs and Chief Patent Counsel. "With these new patents, we have positioned ourselves to capitalize on a broad intellectual property estate, which now enables Sirna to pursue multiple RNAi-based therapeutic approaches."

The methodology, invented by Phillip Zamore, Ph.D. professor of biochemistry & molecular pharmacology, and Gyorgy Hutvagner, Ph.D, both of UMMS, provides methods for inhibition of small RNA function, such as microRNA function in vitro and in vivo. Dr. Zamore and his colleagues developed an elegant system of using short pieces of oligonucleotides that bind to the target microRNAs and block their function, thereby modulating target gene expression. These oligonucleotides are referred to as the anti-RISC oligonucleotides. These patents describe methods that can be used not only for advancing RNAi basic research, but also for developing miRNA-based therapeutics.

"This invention by Dr. Zamore and his colleagues represents a powerful approach for modulating miRNA function," said James P. McNamara, Ph.D., Executive Director of the Office of Technology Management at the University of Massachusetts Medical School. "We are pleased to license this technology exclusively to Sirna as we believe the Company is at the forefront of RNAi-based therapeutic development."

The Zamore miRNA patents are solely owned by the University of Massachusetts. Under the terms of the agreement, Sirna has an exclusive worldwide license to these patents for all uses, including therapeutics, diagnostics, and research reagents. The financial terms of the license were not disclosed.

About RNA interference

RNA interference (RNAi) is a natural, selective process for turning off genes. RNAi is triggered by short interfering RNA (siRNA) molecules that engage a group of cellular proteins, known as RISC (RNA induced silencing complex). The RISC guides the siRNA to its target messenger RNA (mRNA, the messenger between DNA and proteins) by complementary base pairing for the targeted break-up of the mRNA, thus halting protein expression or viral replication. The RISC-siRNA-complex binds and cleaves multiple mRNA molecules in a catalytic fashion.

About Micro RNA

RNA interference (RNAi) is also mediated by short naturally occurring double-stranded RNA molecules known as microRNAs (miRNAs). A large number of miRNAs have been identified in mammalian cells and are believed to regulate the expression of genes and viruses. Aberrant expression of miRNAs has been linked with several diseases, and blocking the function of miRNAs can potentially be a powerful means of treating a number of these diseases.

About Sirna Therapeutics

Sirna Therapeutics is a clinical-stage biotechnology company developing RNAi-based therapies for serious diseases and conditions, including age-related macular degeneration (AMD), hepatitis B and C, dermatology, asthma, Huntington's disease, diabetes and oncology. Sirna Therapeutics completed its Phase 1 clinical trial for Sirna-027 in AMD in 2005 and with its strategic partner, Allergan, Inc., expects to move Sirna-027 into Phase 2 clinical trials in 2006. Sirna has selected a clinical compound for hepatitis C virus, Sirna-034, which the Company plans to bring into Phase 1 clinical trials by the end of 2006. Sirna has established an exclusive multi-year strategic alliance with GlaxoSmithKline for the development of siRNA compounds for the treatment of respiratory diseases. Sirna has a leading intellectual property portfolio in RNAi covering over 250 mammalian gene and viral targets and over 200 issued or pending patents covering other major aspects of RNAi technology. More information on Sirna Therapeutics is available on the Company's web site at

About UMMS

The University of Massachusetts Medical School, one of the fastest growing academic health centers in the country, has built a reputation as a world- class research institution, consistently producing noteworthy advances in clinical and basic research. The Medical School attracts more than $174 million in research funding annually, 80 percent of which comes from federal funding sources. UMMS is the academic partner of UMass Memorial Health Care, the largest health care provider in Central Massachusetts. For more information visit

Safe Harbor Statement

Statements in this press release which are not strictly historical are "forward-looking" statements which should be considered as subject to many risks and uncertainties. For example, Sirna currently does not have any clinical drug candidates or programs based on miRNA technology and the development of Sirna-027 and Sirna-034 as well as Sirna's other programs are still at a relatively early stage. All of these programs, and Sirna's ability to obtain milestone and royalty payments under its collaborations, are subject to significant risks and unknowns, are highly contingent upon future successes, and require significant funding. In addition, patent applications may not result in issued patents, and issued patents may not be enforceable or could be invalidated. Other risks and uncertainties include, among others, Sirna's early stage of development and short operating history, Sirna's history and expectation of losses and need to raise capital, Sirna's need to obtain clinical validation and regulatory approval for Sirna-027, Sirna-034 and Sirna's other product candidates, any of which could have negative results, Sirna's need to engage collaborators, Sirna's need to obtain and protect intellectual property, and the risk of third-party patent infringement claims. These and additional risk factors are identified in Sirna's Securities and Exchange Commission filings, including the Forms 10-K and 10-Q and in other SEC filings. Sirna undertakes no obligation to revise or update any forward-looking statements in order to reflect events or circumstances that may arise after the date of this release.


Rebecca Galler Robison, Senior Director, Corporate Strategy, Sirna

Therapeutics, Inc., 303-449-6500

Sirna Therapeutics, Inc.

CONTACT: Rebecca Galler Robison of Sirna Therapeutics, Inc., SeniorDirector, Corporate Strategy, +1-303-449-6500

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